Trial to Evaluate Safety and Tolerability of ALN-TTR02 in Transthyretin (TTR) Amyloidosis

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The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses of ALN-TTR02 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Condition or disease

Intervention/treatment

Phase

TTR-mediated Amyloidosis

Drug: ALN-TTR02

Phase 2

Access to an investigational treatment associated with this study is available outside the clinical trial.More info ...

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Ages Eligible for Study:

18 Years to 85 Years (Adult, Senior)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Body mass index must be between 17 kg/m2 and ≤ 33 kg/m2;

Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;

Males agree to use appropriate contraception;

Diagnosis of TTR amyloidosis;

Adequate blood counts, liver and renal function;

Willing to give written informed consent and are willing to comply with the study requirements.